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RAG-06
- Development stage
- Preclinical
- Lead developer
- Ractigen Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
RAG-06 is a small activating RNA (saRNA) drug candidate developed by Ractigen Therapeutics for the treatment of spinal muscular atrophy (SMA). Its mechanism of action involves stimulating the expression of the SMN2 gene, thereby increasing levels of survival motor neuron protein, which is deficient in SMA patients. The drug is currently in preclinical development and represents a novel approach to treating neuromuscular diseases by upregulating endogenous gene expression through RNA activation (RNAa)[1][3][4].
02
Targets
SMN2 (Survival motor neuron 2)
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